In patients with polycythemia vera (PV), Jakafi® (ruxolitinib) appears to help control disease-related symptoms and improve quality of life. These findings were presented at the 56th American Hematological Society Annual Meeting and Exposition, December 6–9, 2014, in San Francisco, California.
Polycythemia vera is a slow-growing type of blood cancer that belongs to a group of blood disorders called myeloproliferative neoplasms (MPN). In these disorders, the bone marrow cells that produce blood cells develop and function abnormally. In PV the bone marrow makes too many blood cells, particularly red blood cells. These excess cells thicken the blood and can cause complications, such as a risk of blood clots or bleeding. Without treatment, PV can be life threatening and can eventually progress to more serious blood cancers, such as myelofibrosis or acute leukemia. Effective treatment, however, can significantly decrease risks and complications.
The goal of therapyfor PV is to reduce the thickness of the blood and prevent bleeding and clotting. Jakafi has proven effective in the therapyof PV, including in patients with intolerance or resistance to Hydrea® (hydroxyurea), the most common first-line therapyfor patients with high-risk PV. Jakafi works by inhibiting proteins that may play a role in the development of MPNs by causing the body to make the wrong number of blood cells (JAK1/JAK2 proteins).
A current Phase III study, the RESPONSE trial, is comparing outcomes between Jakafi and best available therapy in patients with PV who can’t tolerate Hydrea or are resistant to it. Participants have been assigned to either best available therapy or Jakafi. Best available therapy includes Hydrea, several other drugs, or no medication. In a primary analysis of RESPONSE outcomes, Jakafi more effectively improved clinical signs of disease (hematocrit control and at least a 35% reduction in spleen size) and symptoms in patients with PV. After eights months of study, patients on best available therapy who had not reached desired disease control were allowed to switch to therapy with Jakafi.
In a separate analysis of data from the RESPONSE trial, researchers evaluated the effect of Jakafi on PV-related symptoms and quality of life. Patients reported the severity of symptoms and rated their quality of life using established questionnaires. They scored their symptoms on a scale from 0 to 10 (0 being absent and 10 being worst imaginable). Symptoms measured included: tiredness, itching, muscle ache, night sweats, sweats while awake, vision problems, dizziness, concentration problems, headache, numbness or tingling, ringing in ears, skin redness, and abdominal discomfort and feeling full early. The researchers compare these outcomes between the Jakafi and best available therapy.
At eight months into the study, patients receiving Jakafi had improved quality of life and symptoms compared those receiving best available treatment. Almost half of patients on Jakafi had a 50% or greater improvement in symptoms versus only 5% of those on best available treatment. Quality of life was also improved for patients receiving Jakafi: 46% of this group reported a small, though measureable, improvement in quality of life versus only 10% in the best available therapy group. Furthermore, at the eight-month mark, patients in the Jakafi group were more likely consider their changes in symptoms “very much improved” or “much improved” (67% versus 13%).
According to these findings from the RESPONSE Trial, patients with PV who are resistant to or intolerant of treatment with Hydrea experience better symptom control and improved quality of life when they are treated with Jakafi instead of best available treatment.
 Mesa R, Verstovsek S, Kiladjian J-J, et al. Changes in Quality of Life and Disease-Related Symptoms in Patients with Polycythemia Vera Receiving Ruxolitinib or Best Available Therapy: RESPONSE Trial Results. Program and Abstracts of the 56th American Hematological Society Annual Meeting and Exposition; December 6–9, 2014; San Francisco, California. Abstract 709.
 Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care: (The RESPONSE Trial) ((RESPONSE)). ClinicalTrials.gov website. Available at: . Accessed December 6, 2014.
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